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Kalydeco (ages 2 - 5) & Orkambi (ages 12+) are variations on a new style of drug to fight Cystic Fibrosis (CF) that treat the underlying protein misfire rather than simply treating the symptoms. It is made by a pharmaceutical company that makes you want to take the sort of full body decontamination scrub normally reserved for exposure to anthrax - Vertex Pharmaceuticals.

Vertex are charging a lot for these drugs - over $200,000 per patient per annum. Partly this revenue goes to providing the management of Vertex with $30 million+ paydays (1) but we need to look past that. At least until we have Australian companies moving back into medical research, but I digress.

About Cystic Fibrosis

Cystic Fibrosis (CF) is the most common life threatening genetic condition in Australia. CF affects many of the body’s systems, including lungs and digestion. In cystic fibrosis there is a problem with the protein that controls the movement of salt in and out of the cells. Too much salt in the cells causes mucus to be very thick and sticky and to build up in organs like the lungs. The mucus clogs the tiny air passages in the lungs and traps bacteria. Repeated infections and blockages can cause irreversible lung damage and death. Mucus can also cause problems in the pancreas, preventing the release of enzymes needed for the digestion of food. This means that people with CF can have problems with nutrition. Today, life expectancy for many people with CF has increased to 38 years. (2)

CF sufferers can spend weeks each year in hospital, at an average of $1400 a day (3). Lung and organ transplants are common treatments, and these run out at around $150,000+ each. So this is one expensive disease to treat. That is without even factoring in opportunity cost - the loss of productivity/wealth creation if CF patients were able to earn and contribute taxation across the full working life they are denied. The out of pocket expenses for an Australian CF patient average $25,000 rising to over $40,000 in later years (5). This does not include the cost to the taxpayer of the 200+ tablets required each week to alleviate CF symptoms.

How the PBS system works

Australia has a single-payer pharmaceutical system called the Pharmaceutical Benefits Scheme (PBS). The Government does not decide which drugs are listed, there is an independent committee called the Pharmaceutical Benefits Advisory Committee (PBAC) that does that. They have a long record of being quite wonderful people doing a very good job in drawing the line between medical benefit and cost to the taxpayers.

Now surprisingly the PBS is not a big ticket item, costing around $9 billion last year out of a Federal budget of $400 billion. The way in which PBAC plays hard ball with BigPharma over prices is one reason why we still have an affordable scheme, and indeed why the USA came gunning for the PBS in the Trans Pacific Partnership negotiations. It is a shining light around the world for socialised or single-payer health care.

PBS Cost

So in early December 2016 the PBAC once again refused to list Kalydeco & Orkambi in the PBS, citing a lack of evidence it provides any real benefit against the very high cost. Thanks to Mark Dickenson for this information:

PBAC ruling

Some discussion has centred around an offer to Vertex by the PBAC to list Orkambi as a limited availability drug for patients with very specific versions of the CF mutation. (4)

Where the Turnbull Government has failed

Here IS where our Prime Minister Malcolm Turnbull and our Health Minister Sussan Ley come in. The Government sets where the PBAC must locate that "trade-off point" between cost and benefit by setting an annual budget for the PBS. An increase in the annual budget moves the trade-off out to include drugs like Orkambi & Kalydeo. The Government also has the discretion to allow a drug to go into widespread use as a trial until the dollars are sorted. Prime Minister Turnbull and Health Minister Ley have chosen to do neither.

We have instead chosen to use the money to send troops back to the never ending war in Syria. My point - smaller government, less wars that we ourselves start to enrich the corporate war machine and more saving of lives right here please Prime Minister.

Let's hear from the young people using Orkambi

The young lady in the image for this story is Ru Aimee. Her Twitter is here. Her thoughts are clearly expressed in that tweet and in her timeline. Thanks for the image permission Ru.

Brisbane sufferer Jessica Bean, 28, participated in the Australian trials and noticed her lung function improve within a day. Ms Bean said the drug turned her life around.

“My therapy has reduced from up to six hours to between one and two hours a day and I'm no longer spending up to six months in hospital,” she said.

Reacting to today’s announcement, Ms Bean said the fight was far from over.

“I’ve had friends who have passed away waiting for Orkambi” she said.(6)

Kate Marshall's testimony to the US Congress hearing into Orkambi was powerful and in the end persuasive, the USA now covers this drug.

Here are a few more testimonials from young people on their Orkambi treatment

I appreciate our system does indeed run a ruler over the cost of a life by turning human beings into "cost of life years". Consider please, Malcolm and Sussan that the allowance for never ending suffering, despair and lives denied does not count for enough in your equation.